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Safety and Efficacy of BAF312 in Dermatomyositis

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ClinicalTrials.gov Identifier: NCT02029274
Recruitment Status : Terminated (The study was terminated prematurely after an interim analysis for futility. The study did not provide any evidence for efficacy of BAF312 in dermatomyositis.)
First Posted : January 7, 2014
Results First Posted : January 15, 2019
Last Update Posted : January 5, 2021
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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Brief Summary:
This study investigated the dose response relationship for the efficacy and safety of BAF312 compared to placebo in active DM patients over a treatment period of 6+6 months and to determine the minimum dose required for a maximal clinical effect. The study was composed of 2 periods: a double-blind period 1 with BAF312 administered at different daily doses (0.5, 2, 10 mg and placebo) and a fixed-dose Period 2 in which BAF312 was administered at the dose of 2 mg daily .

Condition or disease Intervention/treatment Phase
Active Dermatomyositis Drug: BAF312 Drug: Placebo Phase 2

Detailed Description:
The study was prematurely terminated based on the results of an interim analysis where BAF312 did not demonstrate superior efficacy over placebo and a dose-response relationship was not observed. There were no safety concerns. Approximately 56 participants were planned to be randomized. A total of 17 participants were enrolled and randomized by the time the study was terminated.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 17 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Double Blind, Randomized, Placebo-controlled Study to Evaluate, Safety, Tolerability, Efficacy and Preliminary Dose-response of BAF312 in Patients With Active Dermatomyositis (DM)
Actual Study Start Date : August 25, 2013
Actual Primary Completion Date : February 17, 2016
Actual Study Completion Date : February 17, 2016

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Arms and Interventions
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Arm Intervention/treatment
Experimental: BAF312 0.5mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 0.5 mg over a 10 day period. After, participants continued on 0.5 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
 Drug: BAF312
BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
Other Name: Siponimod
Experimental: BAF312 2mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
 Drug: BAF312
BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
Other Name: Siponimod
Experimental: BAF312 10 mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 10.0 mg over a 10 day period. After, participants continued on 10.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
 Drug: BAF312
BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
Other Name: Siponimod
Placebo Comparator: Placebo
During period 1, participants received matching placebo daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
 Drug: Placebo
Matching placebo to BAF312 as tablets for oral administration.


Outcome Measures
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Primary Outcome Measures :
  1. Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score [ Time Frame: Baseline, 6 months ]
    Each muscles tested was evaluated on a 0 - 10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score. The total MMT24 score ranged from 0 - 240, where an increasing trend in the values indicates improvement. A positive change from baseline indicates improvement.


Secondary Outcome Measures :
  1. BAF312 Plasma Concentration [ Time Frame: 6 months ]
  2. Peripheral Blood Lymphocyte Counts [ Time Frame: baseline, 6 months ]
    Absolute lymphocyte counts

  3. Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score [ Time Frame: baseline, 3 months ]
    Each muscles tested was evaluated on a 0-10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score. the total MMT24 score ranged from 0-240, where an increasing trend in the values indicates improvement. A positive change from baseline indicates improvement.

  4. Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test [ Time Frame: baseline, 6 months ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

Written informed consent must be obtained before any assessment is performed.

  • Patients who have been defined as "definite" or "probable" based on the criteria of Bohan and Peter (Bohan and Peter 1975) for dermatomyositis at least 3 months before screening
  • Patients must have active disease as defined by muscle weakness
  • Patients may be on a stable dose of corticosteroid (up/equal to 20 mg once daily prednisone equivalent)
  • Patients currently treated with oral or subcutaneous MTX must have been a stable dose of no more/equal to than 25 mg per week
  • Patients currently treated with Azathioprine must have been a stable maintenance dose of no more/equal to 3 mg/kg/day
  • Negative cancer screening conducted in the 12 months prior to screening visit

Key Exclusion Criteria

  • Dermatomyositis patients having overlap myositis or any other type of myositis including paraneoplastic myositis, drug-induced myopathy, necrotizing myositis
  • Preexisting severe cardiac or pulmonary conditions, malignancy of any organ system or significant eye diseases.
  • Uncontrolled diabetes mellitus or diabetes complicated with organ involvement.
  • Pregnant or nursing (lactating) women
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02029274


Locations
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United States, Arizona
Novartis Investigative Site
Phoenix, Arizona, United States, 85028
United States, California
Novartis Investigative Site
Los Angeles, California, United States, 90095
Novartis Investigative Site
Orange, California, United States, 92868
United States, Florida
Novartis Investigative Site
Miami, Florida, United States, 33136
United States, Kansas
Novartis Investigative Site
Kansas City, Kansas, United States, 66160
United States, Massachusetts
Novartis Investigative Site
Boston, Massachusetts, United States, 02115
Czechia
Novartis Investigative Site
Prague 2, Czech Republic, Czechia, 128 50
Japan
Novartis Investigative Site
Chiba-city, Chiba, Japan, 260-8712
Novartis Investigative Site
Sendai city, Miyagi, Japan, 980-8574
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Additional Information:

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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02029274    
Other Study ID Numbers: CBAF312X2206
2013-001799-39 ( EudraCT Number )
First Posted: January 7, 2014    Key Record Dates
Results First Posted: January 15, 2019
Last Update Posted: January 5, 2021
Last Verified: December 2018
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Dermatomyositis
Additional relevant MeSH terms:
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Dermatomyositis
Polymyositis
Myositis
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Connective Tissue Diseases
Skin Diseases